Latest News: Research
29 September 2017 / Posted in: Research
The request to include those with more than two SMN2 copies and over 7 months of age in the SMA Type 1 nusinersen EAP is not yet successful. We are still waiting to hear from NICE about next steps.
27 September 2017 / Posted in: Research
Novartis has released the following community update for the LMI070 (branaplam) clinical trial.
19 September 2017 / Posted in: Research
Roche have provided an update on clinical trials of their SMN2 splice-modifying drug RG7916
30 August 2017 / Posted in: Research
You can now read the full workshop report written by the clinical researchers and patient representatives from Europe and the US who met last year.
24 August 2017 / Posted in: Research
Sent on 23rd August from clinicians and advocacy groups, this asks for the age limit and SMN2 copy number restrictions on potential access to be removed.
16 August 2017 / Posted in: Research
Roche has begun to enrol SMA patients in the FIREFISH Phase II trial of their splice-modifying drug, RG7916 (also known as RO7034067).
07 August 2017 / Posted in: Research
We have clarified that, from now on going forwards (until we hear from NICE), children will have to be diagnosed by 7 months of age to be eligible. If you have a child who has already been diagnosed with SMA Type 1 who is older than this and not already on the EAP, please read more.
04 August 2017 / Posted in: Research
All hospital costs for administering nusinersen to patients with SMA Type 1 will be provided by NHS England.
21 July 2017 / Posted in: Research
Held on 18th July, this covered the current provision of nusinersen for infants with SMA Type 1 through the Expanded Access Programme (EAP) and what progress there has been towards the possibility of wider access.
13 July 2017 / Posted in: Research
They state that this will be for ‘the remainder of the year in the UK’ and that they will ‘re-evaluate the progress of the reimbursement progress and the EAP towards the end of 2017’.